Memorandum by CancerBACUP (NC 26)
CancerBACUP is the leading national charity
providing information and support to people affected by cancer.
The charity's specialist cancer nurses answer 65,000 enquiries
a year from patients and carers on all aspects of cancer and its
treatment. CancerBACUP's services include a telephone helpline,
a wide range of booklets and factsheets, an interactive website
and a network of local information centres. In addition to providing
information and support, CancerBACUP works to promote patient-centred
services and equitable access to effective treatments.
CancerBACUP welcomed the creation of the National
Institute for Clinical Excellence (NICE), in the belief that national
guidance on the use of particular treatments and guidelines on
the management of specific diseases would improve the quality
of patient care and end unacceptable geographical variations in
access to treatment. We welcome the opportunity to contribute
as stakeholders to NICE technology appraisals and guideline development.
This memorandum will focus on technology appraisals,
as this is the aspect of NICE's work with which we are most familiar.
Cancer treatments have featured prominently in NICE's work programme,
and CancerBACUP has contributed to 11 separate appraisals to date.
We have had a unique opportunity to present the patient's perspective
to NICE, and as a result have acquired a broad insight into how
NICE works and how it fulfils its remit of ensuring that patients
have faster access to modern treatment.
To date NICE has had a positive impact on the
treatment and care received by people with cancer. However, we
have a number of concerns relating to the transparency of the
appraisal process; the absence of adequate data on quality of
life issues; delays in issuing guidance which result in persistent
problems with access to treatment while appraisals are taking
place; and the inconsistency with which NICE guidance has been
implemented to date. We also consider that the NICE process cannot
in itself resolve the wider issue of ensuring sufficient funding
for cancer services.
1. Greater transparency is needed on how
particular treatments are selected for appraisal by NICE, how
the Appraisal Committee reaches the decisions that determine the
guidance it issues to the NHS and how the Committee evaluates
different types of evidence.
2. To help inform NICE's decision-making,
systematic research should be undertaken on patient priorities
and issues relating to quality of life. NICE currently relies
on patient organisations to provide information that should be
collected systematically within clinical trials or as part of
the NHS R&D programme.
3. The appraisal process should be faster,
with shorter gaps between each stage of the process. In particular,
if an appeal is upheld, the guidance should be redrafted immediately,
rather than allowing months to elapse.
4. The Secretary of State should provide
clearer guidance on whether particular treatments should be available
to NHS patients during the appraisal process.
5. Implementation of NICE guidance should
be monitored by the Department of Health, to ensure that NICE
fulfils its remit of promoting faster access to treatment. This
information should be made public.
1. Transparency of the appraisal process
1.1 The clarity and credibility of the guidance
NICE issues to the NHS would be enhanced if the appraisal process
were more transparent. A fully transparent process is vital also
to ensure that the resulting guidance is locally owned and therefore
1.2 Organisations with an interest in the
technologies referred to NICE for appraisal are invited to become
stakeholders in the appraisal process. As a stakeholder, CancerBACUP
has contributed to the process by providing written evidence to
NICE, attending part of the Appraisal Committee's initial discussion
of various technologies, responding to draft guidance produced
by the Committee, and appealing against the Committee's final
decisions on two occasions.
1.3 Patients and carers have an interest
in which treatments are referred to NICE for appraisal, and which
are excluded. However, it is only once a particular technology
has been referred to NICE by the Department of Health or the National
Assembly for Wales that outside organisations are invited to contribute.
At present it is not clear why particular treatments are referred
and others are not. Some treatments NICE appraises are newly licensed,
but others that are referred for appraisal have been available
and in use by the NHS for a considerable time. The process by
which technologies are selected for referral to NICE should become
more open and transparent, so that a broader range of individuals
and organisations have the opportunity to influence NICE's agenda
and thereby influence national guidance on the use of NHS resources.
1.4 Greater transparency is needed from
NICE on the criteria used by the Appraisal Committee in reaching
their decisions. For each appraisal, NICE commissions an independent
assessment report undertaken by a health technology assessment
centre. It should always be possible to see the connection between
the conclusions drawn in the Assessment Report commissioned by
NICE for each appraisal and the final guidance issued by the Committee.
It would be helpful, not only for stakeholders involved in the
process but also for health professionals tasked with implementing
NICE guidance, if the guidance document stated explicitly which
piece of evidence influenced each decision.
1.5 Transparency is also needed in the process
by which expert medical advisers are selected to advise the Appraisal
Committee on particular appraisals. These advisers appear to play
a crucial role in shaping the Committee's views, but it is not
clear how the decision is taken on which experts should be invited.
NICE has recently indicated that stakeholders can nominate expert
advisers, but the process remains ad hoc, with some appraisals
advised by two or three specialists in the field and others by
1.6 It would be helpful if the Committee
provided stakeholders with detailed information about how the
evidence is evaluated. Some organisations, including CancerBACUP,
have been invited to contribute to a number of appraisals without
having a clear idea of what sort of evidence is useful to the
Committee. Given that many patient organisations invest considerable
resources in contributing to NICE appraisals, it is reasonable
to expect some feedback from NICE on whether, and in what way,
the patient's perspective influences the Committee's decisions.
1.7 Another area in which greater transparency
would be welcomed by stakeholders is in definitions of cost-effectiveness
used by NICE. The Appraisal Committee's remit includes assessing
cost as well as clinical effectiveness. It is therefore important
to understand the basis on which cost-effectiveness decisions
are made. It is generally believed that NICE applies a cost-per-QALY
threshold of £30,000. However, this figure does not appear
to be consistently applied and has not been made available to
stakeholders and the wider public. NICE needs to clarify whether
this threshold exists and, if so, how it was determined.
1.8 Related to transparency is the need
to ensure that appraisal guidance sets out explicitly what NICE
intends, in order to avoid a situation where local funders interpret
guidance too narrowly. For example, if NICE recommends that a
particular treatment should not be "routinely used",
the guidance either needs to state clearly in what circumstances
the treatment could be considered, or it should state that clinicians
should have the option to prescribe it. If it does not do this,
local funders could decline to fund the treatment in any circumstances.
2. Research on patients' priorities and quality
2.1 If NICE is to provide guidance that
is clear and credible - not only to the NHS which has to implement
it but to patients who will be affected by itit needs to
base this guidance on high-quality information that accurately
reflects the views and experiences of patients. This information
is not always readily available.
2.2 Most of the cancer treatments referred
to NICE for appraisal offer significant benefits to patients in
terms of quality of life, and many also offer additional survival
time for people with advanced cancer. However, for people with
advanced cancer, quality of lifeby which we mean the extent
to which people with cancer are able to continue with their usual
activities free of pain and other symptoms associated with the
diseaseis often the most important objective and may matter
as much if not more than extra weeks or months of life.
2.3 Guidance issued by NICE suggests that
the Appraisal Committee considers quality of life to be an important
factor in determining which treatments should be recommended for
use by the NHS. However, the experience of cancer appraisals has
highlighted the absence of quality of life data in many clinical
trials. The Committee does not always have available the evidence
it needs to enable it to issue a positive recommendation to the
2.4 One option, in the absence of suitable
data from phase III randomised controlled trials (RCTs), would
be for NICE to give more weight to other types of evidence. At
present, NICE is reluctant to approve treatments that are not
reinforced by data from RCTs. However, it is important that the
Appraisal Committee acknowledges that it may be unethical to ask
patients to take part in randomised trials if the drug being trialled
is the only treatment available for a particular condition. For
many types of advanced cancer, the only alternative to a new drug
is best supportive care. Patients should have the opportunity
to receive treatments that offer them extra survival time or a
better quality of life.
2.5 A more satisfactory long-term option
is to require clinical trials to be designed to take account of
quality of life. Much of the information that would be useful
to NICE in formulating its guidance is not available, as clinical
trials do not routinely collect data on the impact of a particular
treatment on a patient's quality of life. In future we would hope
to see cancer trials designed to collect quality of life data
that both reflects the patient's true experience and satisfies
NICE's need for systematic information on how particular treatments
affect this experience.
2.6 There may also be a role for the NHS
R&D programme in conducting research on the patient's perspective
on treatments for advanced cancer and other diseases, and on quality
of life issues more broadly. NICE says that it genuinely wants
the patient's perspective and sees this as central to the appraisal
If this is the case, NICE may wish to consider commissioning independent
research on patients' views and experiences at the same time as
it commissions the independent Assessment Report for each appraisal.
2.7 A further option would be for NICE to
ask the independent health technology assessment centres that
compile assessment reports to take account of submissions from
stakeholders when summarising the evidence on a particular treatment.
At present the centres only consider published data, and submissions
from stakeholders, including patient organisations, are considered
separately by the Appraisal Committee.
2.8 In the absence of systematic data on
quality of life, patient organisations such as CancerBACUP have
sought to fill in the gaps by providing the Appraisal Committee
with an insight into the views and experiences of patients. However,
this is a limited approach. It is very difficult for patient organisations
to conduct an independent scientific assessment of the impact
of particular treatments on the patients who receive them. If
NICE considers patient organisations to be the most appropriate
source of such information, there should be a system of reimbursement
for these bodies. Reimbursement could come directly from the Department
of Health, if NICE does not have the necessary resources.
3. Timeliness of guidance
3.1 It is vital that NICE appraisals are
completed as quickly and efficiently as possible, to minimise
uncertainty for patients and ensure that patients genuinely have
faster access to effective treatments.
3.2 While some appraisals last longer than
originally envisaged because of a need to consider further evidence,
lengthy delays have occurred for other reasons. It is of particular
concern that the time between an appeal being upheld and the guidance
being redrafted can be so long. During this time no further evidence
can be submitted and patients are often unable to receive treatment
(see section 4 below).
4. Access to treatment during the appraisal
4.1 NICE plays an important role in ending
confusion on the use of the treatments it appraises and in promoting
faster access to these treatments for patients. However, this
positive impact is undermined by the long delays in issuing guidance
on some treatments.
4.2 One of the reasons NICE was created
was to speed up patients' access to effective modern treatments.
There are widespread expectations, not least within Government,
that NICE guidance will resolve the problem known as "postcode
prescribing", whereby access to particular treatments depends
on where patients live and the decisions made by local health
4.3 However, there is a real issue about
what happens while an appraisal is underway. Individual appraisals
can last for a year or more, and there is much anecdotal evidence
that some local commissioners do not fund treatments being appraised
by NICE while an appraisal is ongoing. Far from speeding up access
to treatment, the fact that particular treatments are referred
to NICE appears to sow doubt in the minds of local funders about
whether a treatment is worth funding. Patients have contacted
CancerBACUP's telephone helpline to say that their oncologist
wishes to prescribe a particular treatment but cannot do so because
the treatment in question is being appraised by NICE and funds
are not available until the guidance has been issued. Examples
of these treatments are trastuzumab for breast cancer and irinotecan
for colorectal cancer. This situation has become known as "NICE
blight" and is acknowledged by the Department of Health,
which has recently issued guidance to the NHS that a treatment
for chronic myeloid leukaemia should not be denied to patients
on cost grounds while NICE completes its appraisal.
4.4 In spite of guidance to the NHS from
the Secretary of State that NICE guidance should not be preempted
a number of local commissioners have made clear to clinicians
that funds will not be released for specific treatments unless
and until NICE has given the go-ahead that they should be used.
In the case of cancer, this means that patients are being denied
treatments that their doctors want to prescribe and that may offer
them extra survival time and improved quality of life. People
with advanced cancer are not in a position to wait for NICE to
finalise its guidance, and until guidance is issued clinicians
should be able to prescribe treatments that they believe to be
in the best interests of their patients. It would be helpful if
the Secretary of State clarified and strengthened his previous
guidance to the NHS to this effect.
5. Implementation of guidance by the NHS
5.1 NICE will only make a real difference
to patients if the guidance it issues is implemented effectively.
While the Government's recent announcement that NICE guidance
will become mandatory for health authorities and primary care
trusts from January 2002 is welcome, effective implementation
will not take place unless the guidance is locally owned throughout
the NHS. Greater transparency and stronger data should help promote
local ownership. In the meantime, the announcement that guidance
will be mandatory should help ensure more equitable access.
5.2 An independent survey of health authorities
in England and Wales commissioned by CancerBACUP and carried out
in October and November 2001 demonstrates why it is necessary
that guidance becomes mandatory.
Our research found that local ownership of national guidance is
still some way from being achieved in many areas. Health authorities
expressed the view that compliance with NICE guidance risks distorting
local priorities. Others suggested that NICE guidance raises expectations
among patients which are difficult to fulfil.
5.3 CancerBACUP's survey found that NICE
guidance is not being implemented uniformly throughout the NHS.
While some health authorities have set aside funds to ensure that
NICE guidance is fully implemented, the majority have not. Fewer
than half the health authorities in England and Wales have a policy
for monitoring local compliance with NICE guidance, and most do
not know whether all suitable patients are being offered treatments
recommended by NICE.
5.4 The survey also showed that it takes
many months for NICE guidance to be fully implemented. While most
health authorities are implementing NICE guidance on taxanes for
breast and ovarian cancer issued in the summer of 2000, far fewer
are implementing guidance issued more recently on treatments for
other cancers. It is vital that the Secretary of State clarifies
whether the NHS will be expected to implement guidance already
issued as well as guidance issued after January 2002.
5.5 It is now imperative that the Department
of Health monitors effectively the extent to which local health
commissioners implement and fund national guidance. Systematic
monitoring is needed and the results should be placed in the public
domain to enable patients and the wider public to see what impact
NICE is having on the treatment and care patients receive in practice.
22 Quality-Adjusted Life Year. Back
NICE, Guidance for patient/carer groups (June 2001). Back
NICE, Faster access to modern treatment (1999). Back
Department of Health, Health Service Circular 1999/176 (6 August
CancerBACUP, Implementing NICE guidance on treatments for cancer
(December 2001)-see Appendix attached. Back